Vertex Pharmaceuticals Incorporated, a global biotechnology company, announced that Health Canada has approved PrOrkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation. It is only indicated for these patients, who can be identified with a genetic test.

Cystic fibrosis is a rare, life-threatening genetic disease. People with two copies of the F508del mutation represent the largest group of people with CF. Of the approximately 4,000 people in Canada with CF, approximately 1,500 of these people ages 12 and older have two copies of the F508del mutation.

"With Health Canada's approval of Orkambi, people in Canada living with the most common form of CF now have a treatment for the underlying cause of the disease," said Andy Partridge, Vertex vice president and head of the North American Commercial Organization.

The approval of Orkambi was based on data from two phase 3 studies (TRAFFIC and TRANSPORT) that enrolled more than 1,100 people with CF ages 12 and older with two copies of the F508del mutation.

Vertex continues to invest in CF research and development, including at our Laval, Quebec facility, with the goal of developing treatments for the vast majority of people with the disease. Multiple phase 2 and phase 3 clinical studies are in progress.

"Like PrKalydeco (ivacaftor) before it, Orkambi treats the underlying defect in cystic fibrosis, but for a much larger part of the CF population. Health Canada's approval of Orkambi is the first step in getting this drug into the hands of the people who need it most, the indicated patients for whom Cystic Fibrosis Canada advocates each and every day," said Cystic Fibrosis Canada Chief Scientific Officer Dr. John Wallenburg. "We congratulate Vertex on the approval of Orkambi by Health Canada and look forward to working with Vertex and the provincial drug plans to ensure access to Orkambi for eligible CF patients across Canada."

Health Canada approval is the first step in the process for securing funding through Canada's public drug programs for a new medicine. Once a new medicine receives Notice of Compliance (NOC), or approval from Health Canada, it enters the Canadian Agency for Drugs and Technologies (CADTH) Common Drug Review (CDR) process. Following its review, CADTH will send a recommendation to participating public drug programs. In Quebec, new medicines are reviewed by l'institut national d'excellence en santé et en services sociaux (INESSS). Participating jurisdictions may then proceed to reimbursement discussions with the manufacturer through the Pan-Canadian Pharmaceutical Alliance (pCPA). When an agreement has been reached between the manufacturer and the pCPA, each province and territory determines how the new medicine will be funded.

Cystic fibrosis is a rare genetic disease that is caused by defective or missing cystic fibrosis transmembrane conductance regulatory (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little to no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Orkambi is a combination of lumacaftor, which is designed to increase the amount of protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Orkambi is recommended to be taken every 12 hours - once in the morning and once in the evening.

Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives.